Narcolepsy Community Sets Record Participation in FDA’s Patient-Focused Initiative

by julie on September 28, 2013

“We are a bunch of deeply motivated people doing everything in our power to live full lives.”

– Saraiah, Narcolepsy Advocate

Leading up to the FDA Narcolepsy Meeting on Sept. 24, 2013, I traveled to FDA’s campus four times, wrote 15 blog posts, co-led the Unite Narcolepsy initiative, co-presented webinars, wrote FAQs, and coached panelists – yet nothing could have prepared me for this day.

Just before 1:00 p.m., the Great Room at FDA buzzed with excitement and nerves, as 150 narcolepsy patients, caregivers, and stakeholders filled the room to capacity. At 1:00 p.m., the room fell silent as FDA representatives welcomed the narcolepsy community and provided introductory remarks. Another 700 narcolepsy advocates joined us via webcast from around the world. I smiled from ear-to-ear, proud to have helped secure this opportunity for our community.

Narcolepsy is the fourth disease area addressed by FDA’s ground-breaking Patient Focused Drug Development Initiative and the first rare disease to go through the process. The initiative aims to better understand patients’ perspectives on symptoms, severity, and current treatment options – to help FDA make more informed regulatory decisions when assessing the risks and benefits of future therapies.

Topic 1: Narcolepsy symptoms and daily life impact

The first five patient panelists took their seats at the front of the room to kick off the Topic 1 discussion on narcolepsy’s symptoms and daily life impact. Each panelist eloquently described his or her journey with narcolepsy.

One young woman, Kerry visited 20 – 25 specialists before finding an accurate diagnosis. In the process, she was misdiagnosed with epilepsy and put on heavy drugs for a condition that she did not have. Doctors encouraged her to leave school, but she has persisted.

Another panelist, Carrie poignantly stated, “The limitations on my time and energy affect my ability to give to society, to my kids, and to finish projects. There is so much inside that I want to do and create.”

My throat clenched and eyes welled with tears. I did not expect these emotions, but the discussion hit a nerve – reminding me of the frustrations of delayed diagnosis and the serious life-changing nature of narcolepsy. After the panelists’ testimony, the group discussion began including audience and webcast participation.

The FDA representatives did an amazing job facilitating this highly emotional discussion with kindness, respect and professionalism. One fellow patient, Saraiah recalls, “I was deeply impressed with the skill that the FDA staff brought to facilitating that meeting and helping to communicate back to us that we were heard.”

Topic 2: Narcolepsy Treatment

After a short break, Topic 2 panelists provided testimony on current treatment options. Panelists addressed a wide range of medications, alternative treatments, side effects and complications. No two people’s experience was exactly the same, but our collective determination to feel better and make the most of our lives was very apparent.

A lively discussion followed with one patient clearly stating, “A nap is not a treatment, it’s a symptom. It’s uncontrollable.” The audience broke into applause. FDA listened closely and took copious notes.

For future treatments, the aspects of narcolepsy that patients would MOST like addressed are EDS, brain fog, sleep architecture and hypocretin deficiency. The meeting closed with an open-mic portion and summary from FDA.

In Closing:

Afterwards, Unite Narcolepsy hosted a reception to celebrate our community’s accomplishments. It was wonderful to see many familiar faces and make new friends.

Thank you to the panelists, attendees and webcast participants who gave their time and energy to this meeting. We “wowed” the FDA and the rare disease community with our record-setting participation.

Personally, I will never forget this day. Fellow advocate, Saraiah said it best, “The overall message of the meeting was that we are a bunch of deeply motivated people who are doing *everything* in our power to live full lives.”

I cannot thank you enough for all your efforts to make this event happen. My son and I watched most of the meeting through web conferencing. He was so excited to see familiar PWN faces and wanted me to submit comments too. I told him that I would if I felt like there was ever a point that the folks in the meeting did not address, and I never found one! My son was disappointed because he wanted me to make a comment, and I was happy to have been represented by an awesome group. Thanks to everyone in attendance who made every point I would have wanted to make and many more!

Thank you for reading my post, Jill. I’d hoped to get my re-cap done sooner, but the meeting was so emotional and moving, it left me speechless for a few days. I’ll share the links to the video and transcript on my blog as soon as possible.

Hi Julie, Thank you for all you do it is amazing. It never ceases to amaze me as a care giver to hear how this disorder affects everyone. Always the same symptoms but always in different ways. The severity of it for some is just heart wrenching and I have to say that it was a good thing I was participating through the web because I cried through the whole thing and that would not have been a pleasant site. I am grateful that my son’s narcolepsy is not as severe as some and he can have somewhat of a normal life this is driving me and him to do more for narcolepsy awareness. Thank you again you are truely an amazing person!

Thank you for your support, Michele. I cried through most of it too, and there were many sniffles and waivering voices all around me so you were in company! We are incredible people overcoming so much adversity daily. So very proud of our eloquent, well-spoken strong community. Thank you for supporting your son and all of us with narcolepsy.

Thank you! I had to miss the webcast as it was 3am here in Australia and I had my first sleep study the night before: cue exhaustion.

I can’t wait to watch when it comes out! I would have leapt to my feet in applause at that nap comment. I am yet to have any doctor tell me to nap because they haven’t yet decided my problem, but I CAN’T nap. Once asleep it always lasts at least an hour instead of the suggested 20 minutes, and I ALWAYS feel like death afterwards. It’s just not worth it!

I’m also very pleased that the “would most like addressed” list pretty much matches what I want addressed. Good work everyone!

Thank you so much for reading my post, Danielle and taking an interest in our FDA initiative all the way from Australia!! I always feel like death after my naps too – I’m going to write an upcoming post about it on my blog. Thanks again for your support, Julie

I really look forward to seeing more…. thanks.
I’m really excited about you Julie. About your impact. It’s really awe inspiring. You’re like a home run hitter. The bases were loaded — with years and years of efforts and organizing in the community, for which I am very proud to have helped create — but you’ve brought it all home! Our home run hitter.
Great respect and gratitude. And awe.

As a Respiratory Therapist diagnosed with N in March 2012, I suffered through school & many years prior. I love your book & wished I could’ve made the FDA conference. Thanks to you & all you do from day to day.
Hope to meet you someday.
GOD Bless,
Amber

Thank you Julie and to all who attended the session and shared their stories! I too was incredibly moved, emotional, and yes still somewhat frustrated to hear of the many ways Narcolepsy can impede the most basic of daily functioning! I am a mom of a 17 year old Narcoleptic daughter who has been diagnosed since age 9. Life for us continues to be a trial and error of drug treatments which sometimes have helped greatly and other times caused very serious life threatening side effects. I know that with all of our collective effort and continued push for change that we can see this disorder one day (hopefully soon) have a cure and not just treatments!!! Together we are stronger then we realize. I am so very thankful for this online community which reaches around the world and can unite us in our struggles, joys, and continued advocacy!!! We are NEVER ALONE and we can be the change that is NEEDED!! Keep UP the AWSOME WORK and THANK YOU AGAIN JULIE FOR ALL THAT YOU DO FOR PWN!!!

Beth K. Thank you for supporting your daughter’s experience with N and my mission of building a brighter future for all those with this condition. This means the world to me. I cannot even begin to express my gratitude. Thank YOU for your support, Julie

Thank you, Ms. Sara! That means so much to me coming from a great writer and editor like yourself. As you guessed, it wasn’t quite as effortless to write this as it may appear. I spent many hours writing this, as I wanted so badly to capture the energy in that room – perhaps an impossible task. SO great to see you there and looking forward to seeing you again in a few weeks!